NCCN OKs JAK Inhibitor in Myelofibrosis Treatment

Treatment guidelines are 'urgently needed'

ORLANDO – Treatment of intermediate-risk myelofibrosis should be with ruxolitinib (Jakafi), hematopoietic cell transplantation (HCT), or enrollment in a clinical trial, according to guidelines from the National Comprehensive Cancer Network (NCCN).

In the new guidelines, NCCN committee chair Ruben Mesa, MD, of the Mayo Clinic in Phoenix, and colleagues also stated that observation with regular assessments may be an acceptable treatment avenue in this patient population.

"These treatment guidelines for myelofibrosis are ... most urgently needed in terms of guidance," Mesa said at the NCCN meeting.

While myelofibrosis is a rare disease, its chronic nature means that people can live with it for decades.

"It is a bit surprising that we are just getting around to writing guidelines for these diseases," said James Gallagher, MD, of the Geisinger Health System in Danville, Penn. "We don't diagnose many of these patients each year, but there are quite a number of them in the community because these patients live a long time with this disease. These guidelines are needed."

For low-risk myelofibrosis, the guidelines recommend the following:

Asymptomatic: observation or clinical trial; monitor for signs or symptoms of disease progression every 3-6 months

In patients who do not respond or show loss of response, treatment with ruxolitinib, interferons, or clinical trial can be revisited, according to the guideline.

For patients with high-risk disease, treatment courses include clinical trial (platelets ≤50,000), or ruxolitinib or clinical trial (platelets ≥50,000). Depending on their risk score, those with high-risk myelofibrosis may also be candidates for allogeneic HCT.

The guidelines suggest that patients who have progressive disease after various treatments should also be considered for transplant. If they turn out to be not candidates for transplant, they should be enrolled in a clinical trial or follow guidelines for acute myeloid leukemia.

He noted that differences in mutations in myelofibrosis may substantially impact prognosis. For example, a patient without mutations can expect a life expectancy of about 17 years versus around 8 years for one with a mutation.

Mesa said that the next set of guidelines for other myeloproliferative neoplasms, such as polycythemia vera and essential thrombocythemia, are in the final stages and may be added to the NCCN compendium later in 2017. After that, the committee will set about to write guidelines for atypical myeloproliferative neoplasms.

"There are not great numbers of these patients but their treatment is distinct and there is no clear guidance for them," he said of the latter.

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